A pivotal moment in the life of a potential new medicine is the period of gradually-expanding tests on human volunteers known as clinical trials. Clinical trials are vital to determining the impact of a new potential treatment. With strict oversight by Institutional Review Boards, clinical trials provide the U.S. Food and Drug Administration with the scientific information needed to weigh the benefits and risks of a new medication and decide whether it is safe for patients.

What Are Clinical Trials?

Once researchers have completed a rigorous screening and preclinical testing process, the company files an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA). This application allows the investigational drug to be tested in human volunteers in clinical trials. A clinical trial is a carefully designed study which tests the benefits and risks of a specific medical treatment or intervention, such as a new drug or a behavior change (e.g., diet).

Every clinical trial is led by a principal investigator, who is usually a doctor, along with a team of nurses and others researchers. The FDA requires a multi-phase clinical trials process to be completed before deciding if the medicine under investigation is safe and effective for a broader patient population. Usually, these number of human volunteers in the trial increases as the treatment moves through these phases. That is why innovative medicines cannot be developed without the help of volunteers who participate in clinical trials.

How Are Drugs Discovered?

Developing a new medicine begins with understanding the disease or condition as thoroughly as possible. Basic research provides clues about how to treat diseases and potential ways to target the symptoms or underlying causes. Armed with an idea, researchers work to understand biological targets for a potential medicine. A “drug target” can be a protein, RNA, DNA or other molecule that is somehow involved in the disease. Researchers conduct studies in cells, tissues and animal models to determine whether the target can be influenced by a medicine. They then look for a lead compound — a promising molecule that could influence the target and, potentially, become a medicine. This is the first step in the discovery and development process, which spans from initial research to the delivery of life-saving or life-enhancing new medicine.

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The Biopharmaceutical Research & Development Process

From drug discovery through FDA approval, developing a new medicine takes at least 10 years on average and costs an average of $2.6 billion.* Less than 12% of the candidate medicines that make it into Phase 1 clinical trials will be approved by the FDA.

FDA Review & Approval

If the results of all required clinical trials phases show that the investigational new drug is safe and effective, the company submits a New Drug Application or Biologics License Application to the FDA. This application includes reams of data from all the stages of testing, and is a request for FDA approval to market the new medicine.

Scientists at the FDA carefully review all the data from all of the studies on the drug under investigation and, after weighing the benefits and risks of the potential medicine, decide whether to grant approval. Occasionally, the FDA will ask for additional research before granting approval or even convene an independent expert panel to consider data presented by the FDA and the company. The panel will then advise the agency on whether to approve the application and under what conditions.